Article
Design of efficacious somatic cell genome editing strategies for recessive and polygenic diseases.
Nature communications - 8 Dec 2020
Carlson-Stevermer Jared, Das Amritava, Abdeen Amr A, Fiflis David, Grindel Benjamin I, Saxena Shivani, Akcan Tugce, Alam Tausif, Kletzien Heidi, Kohlenberg Lucille, Goedland Madelyn, Dombroe Micah J, Saha Krishanu
Abstract excerpt
Compound heterozygous recessive or polygenic diseases could be addressed through gene correction of multiple alleles. However, targeting of multiple alleles using genome editors could lead to mixed genotypes and adverse events that amplify during tissue morphogenesis. Here we demonstrate that Cas9-ribonucleoprotein-based genome editors can correct two distinct mutant alleles within a single human cell precisely....
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